血友病A的治疗进展

Update on the Treatment of Haemophilia A

血友病A是一种先天性出血性疾病。治疗目标是防止出血,降低与关节损伤有关的长期并发症的风险,并通过维持适当的因子VIII [FVIII]水平来减少出血并改善生活质量。近年来,血友病的治疗有了巨大的突破。在此,我们对血友病A的治疗进行了回顾,发现替代疗法的改变使血友病患者的出血率显著下降,但抑制剂的问题也日益凸显,此外,各种新型治疗方式也纷纷涌现出来。例如延长半衰期的凝血因子产品、模拟FVIII凝血功能的双特异性抗体和基因治疗等,为血友病的治疗带来新的曙光。同时也讨论了如何更好地从我国实际情况出发贯彻落实血友病综合关怀模式。

Haemophilia A is a congenital bleeding disorder. The therapeutic goal is to prevent bleeding, reduce the risk of long-term complications associated with joint damage, and reduce bleeding and improve the quality of life by maintaining the appropriate level of Factor VIII [FVIII]. In recent years, there have been tremendous breakthroughs in the treatment of haemophilia. Here we review the chang-ing treatment of haemophilia A, the transition of alternative treatment modes evidently decreased the incidence of haemophilia, but inhibitor issues were also gradually highlighted. In addition, var-ious new treatment methods have also emerged, such as extended half-life clotting factor products, dual specific antibodies to mimic the coagulation function of FVIII and gene therapy, bringing a new light to the treatment of haemophilia. At the same time, how to better implement the comprehen-sive care model of haemophilia from the actual situation of our country has also been discussed.